To evaluate the safety and efficacy of QGE031 versus placebo in patients with bullous pemphigoid. Efficacy will be assessed as a reduction of disease activity. How QGE031 is broken down by the body and the impact it has on different blood and tissue markers will also be explored.

A Randomized, Double-blind, Placebo Controlled, Parallel Group Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of QGE031 in the Treatment of Patients With Bullous Pemphigoid With Disease Refractory to Oral Steroid Treatment

QGE031 will be evaluated at various dose levels and regimens, based on the impact on disease of the next highest dose level and regimen.

Placebo Comparator: Placebo

Placebo to Match Q2W s.c.

Drug: Placebo

Placebo will be used to control for normal variability in disease severity.

Experimental: Open Label QGE031

Open Label QGE031 Q2W s.c.

Drug: QGE031

QGE031 will be evaluated at various dose levels and regimens, based on the impact on disease of the next highest dose level and regimen.

Detailed Description:

This study was planned to be divided into 2 distinct parts. Part 1 was a multicenter, randomized, placebo-controlled study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment. Patients were treated with QGE031 or placebo in a 2:1 ratio.

Part 2 of this study was planned to be a multi-center, open label, dose range finding study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment.

This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( >50% better then placebo)

Eligibility

Ages Eligible for Study:

20 Years to 80 Years (Adult, Senior)

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Patients diagnosed with bullous pemphigoid

Stable dose of prednisone at or above 10mg per day but no greater than 1 mg/kg/day

Weigh between 40-120kg

total IgE level up to 5000 IU/mL

Exclusion Criteria:

Use of rituximab within 1 year

Other protocol-defined inclusion/exclusion criteria may apply

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01688882